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Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) is at the forefront of RNA interference (RNAi) therapeutics, pioneering a transformative class of medicines aimed at treating and potentially curing a wide range of diseases. Founded in 2002 and based in Cambridge, MA, Alnylam has translated Nobel Prize-winning science into five commercial products: ONPATTRO (patisiran), AMVUTTRA (vutrisiran), GIVLAARI (givosiran), OXLUMO (lumasiran), and Leqvio (inclisiran), developed and commercialized in partnership with Novartis. These breakthrough therapeutics address conditions such as hATTR amyloidosis, acute hepatic porphyria, primary hyperoxaluria type 1, and hypercholesterolemia.
Alnylam is dedicated to expanding its pipeline with several investigational medicines in late-stage development, focusing on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and CNS/ocular diseases. The company’s commitment to sustainable innovation is reflected in its robust product pipeline and strategic collaborations with industry leaders like Roche and Regeneron.
Financially, Alnylam reported robust growth in 2023 with $1.24 billion in global net product revenues, a reflection of its strong commercial execution across its therapeutic products. The company maintains a strong cash position, bolstered by upfront fees from research partnerships and milestone achievements.
Alnylam is also dedicated to corporate responsibility, with initiatives aimed at reducing greenhouse gas emissions, promoting diversity and inclusion in clinical trials, and expanding global health equity through its Alnylam Challengers program. The company’s environmental impact data is third-party verified, underscoring its commitment to transparency and sustainability.
Looking ahead, Alnylam aims to sustain its leadership in RNAi therapeutics with the anticipated release of topline results from the HELIOS-B Phase 3 study and the continued development of promising candidates like zilebesiran for hypertension and mivelsiran for Alzheimer’s disease. For more details on Alnylam’s people, science, and pipeline, visit www.alnylam.com.
Regeneron (REGN) and Alnylam (ALNY) announced positive preliminary data from a Phase 1 study of ALN-HSD, aimed at treating nonalcoholic steatohepatitis (NASH). Results show robust target knockdown and lower liver enzymes in patients receiving ALN-HSD compared to placebo, with a favorable safety profile. The companies plan to advance to a Phase 2 trial in late 2022. The study highlighted a significant unmet need in NASH, which affects 16 million people in the U.S. and is projected to be a leading cause of liver transplants.
Alnylam Pharmaceuticals (NASDAQ: ALNY) will present new data from the APOLLO-B Phase 3 study of patisiran at the HFSA Annual Scientific Meeting on
Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced the pricing of a $900 million private offering of 1.00% convertible senior notes due in 2027. The offering allows initial purchasers to buy an additional $135 million of the notes. The notes will accrue interest payable semi-annually and will be convertible under specified conditions. Proceeds are projected to be approximately $883.2 million, intended for capped call transactions and to repay existing borrowings. Closing is expected on September 15, 2022, pending customary conditions.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has initiated a private offering of $900 million in convertible senior notes due 2027, with an option for initial purchasers to acquire an additional $135 million. The notes, which are senior and unsecured, will mature on September 15, 2027, and interest will be paid semi-annually. Alnylam plans to use around $762 million of the proceeds to repay existing borrowings and for general corporate purposes. The offering is under Rule 144A and is contingent upon market conditions.
Alnylam Pharmaceuticals announced positive results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic for treating ATTR amyloidosis with cardiomyopathy. The study met its primary endpoint, showing a significant improvement in functional capacity (14.7 meters in 6-MWT) and health status (3.7 points in KCCQ-OS) compared to placebo after 12 months. Safety profiles were encouraging, with no major cardiac concerns. A supplemental new drug application is planned, indicating a potential market opportunity for patisiran if approved.
Alnylam Pharmaceuticals (NASDAQ: ALNY) will present a company overview at the Morgan Stanley 20th Annual Global Healthcare Conference on September 13, 2022, at 11:10 am ET. The event will take place at the Sheraton New York Hotel. Investors can access a live audio webcast on the company’s website, with a replay available within 48 hours post-event. Alnylam is known for pioneering RNA interference (RNAi) therapeutics, addressing rare diseases with innovative products like ONPATTRO and GIVLAARI.
Alnylam Pharmaceuticals announced positive results from a Phase 2 study of cemdisiran for treating immunoglobulin A nephropathy (IgAN). The study demonstrated a 37% reduction in the 24-hour urine protein to creatinine ratio compared to placebo. Notably, 32% of patients on cemdisiran achieved a significant reduction in protein levels, compared to 13% on placebo. The treatment was well tolerated, with common side effects being injection site reactions. Alnylam aims to advance cemdisiran to Phase 3 development, addressing the significant unmet needs in IgAN treatment.
Alnylam Pharmaceuticals has appointed Dr. Elliott Sigal to its Board of Directors, enhancing its leadership at a pivotal moment. With decades of experience, including his role as Chief Scientific Officer at Bristol-Myers Squibb, Dr. Sigal contributed to the launch of 14 new medicines across various therapeutic areas. His expertise is expected to strengthen Alnylam's ongoing efforts in advancing RNAi therapeutics aimed at rare diseases. The company continues its pursuit of transforming healthcare through innovative treatments, guided by its 'Alnylam P5x25' strategy.
Alnylam Pharmaceuticals has announced the presentation of data from the APOLLO-B Phase 3 study of patisiran at the 18th International Symposium on Amyloidosis on
Alnylam Pharmaceuticals announced that the APOLLO-B Phase 3 study of patisiran met its primary endpoint, showing a statistically significant improvement in the 6-Minute Walk Test compared to placebo at 12 months (p-value 0.0162). The study also achieved its first secondary endpoint, indicating improved quality of life via the Kansas City Cardiomyopathy Questionnaire (p-value 0.0397). The company plans to file a Supplemental New Drug Application in the U.S. in late 2022. Patisiran demonstrated a favorable safety profile, with fewer deaths in the treatment arm.